DREAM01

DREAM01 is an ex vivo gene therapy developed by Assistance Publique - Hôpitaux de Paris for the treatment of severe sickle cell disease (SCD). The therapy utilizes a bifunctional lentiviral vector to transduce autologous CD34+ hematopoietic stem cells. This vector is designed to simultaneously express a therapeutic beta-globin variant (βAS3m) and a micro-RNA (miR7m) that specifically targets and silences the endogenous sickle hemoglobin (βS-globin) mRNA. By increasing the production of non-sickling hemoglobin and reducing the levels of sickling hemoglobin, the treatment aims to prevent the polymerization of hemoglobin S, thereby reducing vaso-occlusive crises and long-term organ damage in patients who lack a matched sibling donor.

Other names

Autologous CD34+ cells transduced with βAS3m/miR7m lentiviral vector